Research programme: gene editing therapeutics - CRISPR Therapeutics

Drug Profile

Research programme: gene editing therapeutics - CRISPR Therapeutics

Alternative Names: CRISPR-Cas9 gene therapies

Latest Information Update: 16 Aug 2017

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At a glance

  • Originator CRISPR Therapeutics
  • Developer CRISPR Therapeutics; Genethon; University of Florida; Vertex Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene modulators; Immunomodulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Beta-thalassaemia; Cancer; Haemoglobinopathies; Mucopolysaccharidosis I; Sickle cell anaemia
  • Research Amyotrophic lateral sclerosis; Cystic fibrosis; Dementia; Duchenne muscular dystrophy; Glycogen storage disease type I; Unspecified

Most Recent Events

  • 10 Aug 2017 CRISPR Therapeutics has patent protection for CRISPR-Cas9 genome editing technology in Australia, New Zealand and Singapore
  • 10 Aug 2017 CRISPR Therapeutics announces intention to submit CTA to EMA for Haemoglobinopathies in late 2017
  • 10 Aug 2017 CRISPR Therapeutics plans clinical trials for Haemoglobinopathies in 2018
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